Spark adds fresh ev­i­dence that pi­o­neer­ing gene ther­a­py could be a game chang­er for pa­tients

Spark Ther­a­peu­tics has added an­oth­er im­pres­sive round of late-stage da­ta demon­strat­ing that its gene ther­a­py for an in­her­it­ed eye dis­ease could be a game chang­er for pa­tients. This lat­est read­out in­clud­ed pos­i­tive out­comes for 9 crossover pa­tients who re­ceived voreti­gene nepar­vovec (SPK-RPE65) a year ago, along with promis­ing longer term re­sults on the orig­i­nal group of 20 pa­tients.

Nine pa­tients with in­her­it­ed reti­nal dis­ease caused by mu­ta­tions in the RPE65 gene were put in the con­trol arm of the study, and all vol­un­teered for the gene ther­a­py af­ter in­ves­ti­ga­tors con­clud­ed that the orig­i­nal treat­ment group had ben­e­fit­ed marked­ly with sig­nif­i­cant gains in vi­sion. A year af­ter treat­ment, Spark says that 8 of the 9 “demon­strat­ed the max­i­mum im­prove­ment mea­sur­able on the pri­ma­ry end­point at one year.” And the first 20 pa­tients who were treat­ed with their gene ther­a­py have demon­strat­ed a sus­tained ben­e­fit in vi­sion, speak­ing to the dura­bil­i­ty of the change.

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